The present invention concerns methods of treating subjects afflicted with Batten disease.
Batten disease is a group of neurodegenerative disorders characterized by cognitive decline, spasticity and seizures due to neuronal cell loss, and blindness due to retinitis pigmentosa. The juvenile form of Batten disease is an autosomal recessive neurodegenerative disease of childhood (R.-M. Boustany, et al., Am. J. Med. Genet. Suppl. 5: 47-58 (1988)). It is clinically characterized by onset at age 5-6 years with progressive blindness, generalized and myoclonic seizures, cognitive and motor decline and death in the mid to late twenties (R.-M. Boustany and E. H. Kolodny, Rev. Neurol. (Paris) 145: 105-110 (1989); R.-M. Boustany, Am. J. Med. Genet. 42: 533-535 (1992); R.-M. Boustany and P. Filipek, J. Inher. Metab. Dis. 16: 252-255 (1993)). No suitable treatments for Batten disease are currently available. Therefore, there is an urgent need for the development of new ways to treat Batten disease.
A first aspect of the present invention is a method of treating Batten disease in a subject in need thereof. The method comprises administering to said subject an active agent selected from the group consisting of flupirtine and the pharmaceutically acceptable salts thereof in an amount effective to treat Batten disease in that subject. The administering step is preferably an oral administering step, and the active agent is preferably flupirtine maleate.
A second aspect of the present invention is the use of flupirtine or a pharmaceutically acceptable salt thereof for the preparation of a medicament for the treatment of Batten disease.
Flupirtine (available in some regions in the flupirtine maleate form as the drug Katodolon(copyright) from ASTAmedica) was developed as a centrally acting, non-opiate analgesic. It is currently in use in Germany, Brazil, Italy and other countries. It has been used in adults and children safety and chronically for over 12 months without undue side effect or addiction. It has been shown to counteract apoptosis in retinal pigment epithelial cells and neuronal cells in culture. It has been shown to reduce the area of ischemia induced by stroke in rats pretreated with it. It has been shown to have anticonvulsant effects and very powerful muscle relaxant effects as well. The use of flupirtine in the treatment of Batten disease has neither been suggested nor disclosed.
The present invention is explained in greater detail in the specification set forth below.